Clinical Studies

Ongoing Clinical Studies

We support the disease communities – and their families

Contact us for additional information about the studies listed below at clinicaltrials@amicusrx.com.

Epidermolysis Bullosa (EB)

SD-101: Phase 3 Study SD-005
Study of Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa

  • Status: Ongoing, currently recruiting patients 1 month and older with a diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB who have a wound that meets specific study criteria as assessed by a healthcare professional.
  • Design: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of SD-101 Cream. SD-101 (6%) or placebo will be applied topically, once a day to the entire body for a period of 90 days. Patients who complete the study will be eligible to enroll in an open-label extension Study (SD-006).
  • More information: www.clinicaltrials.gov: NCT02384460

SD-101: Open-Label Extension Study SD-004
Open Label Extension Study to Evaluate the Safety of SD-101 Cream in Subjects with Epidermolysis Bullosa

  • Status: Ongoing, recruitment closed. Patients who completed the treatment period in the Phase 2b study (SD-003) of SD-101 were eligible to roll over into SD-004.
  • Design: Open-label extension study to assess the continued safety of topically applied SD-101 Cream (6%) in subjects with Simplex, Recessive Dystrophic, and Junctional non-Herlitz EB. SD-101 (6%) is applied topically, once a day to the entire body.
  • More information: www.clinicaltrials.gov: NCT02090283

Pompe Disease

ATB200/AT2221: Phase 1/2 Safety Study (ATB200-02 Study)
Open-label, Dose-escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous ATB200 Co-administered with Oral AT2221 in Patients with Pompe Disease

  • Status: Ongoing, currently recruiting patients 18 to 65 years with a diagnosis of Pompe disease.
  • Design: Open-label, dose-escalation study of Pompe disease patients to assess if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.
  • More information: www.clinicaltrials.gov: NCT02675465

Fabry Disease

Migalastat Monotherapy: Phase 3 Long-Term Safety Study 041 (AT1001-041 Study, formerly MGM116041)

Long-Term, Open-Label Study of Migalastat (AT1001) in Subjects with Fabry Disease Who Completed Treatment in a Previous Monotherapy Trial with Migalastat

  • Status: Completed. Patients are eligible to roll over into Study 041 upon completion of the primary treatment and open-label extension periods in the FACETS (Study 011), ATTRACT (Study 012), or Phase 2 Extension Study 205.
  • Design: Open-label extension study to evaluate long-term safety and efficacy of migalastat.
  • More information: www.clinicaltrials.gov: NCT01458119

Migalastat Monotherapy: Phase 3 Long-Term Safety Study 042 (AT1001-042 Study)

Open-Label Extension Study of the Long-Term Effects of Migalastat in Patients with Fabry Disease

  • Status: Recruiting. Intended to provide continued treatment with migalastat for subjects with Fabry disease who completed treatment of a previous migalastat monotherapy study.
  • Design: Open-label extension study to assess the long-term safety and effectiveness of migalastat in subjects with Fabry disease who completed migalastat treatment in a previous study.
  • More information: www.clinicaltrials.gov: NCT02194985

Migalastat Monotherapy: Physician Initiated Request (MGM116188)

Program Allows Physicians to Request Permission from Amicus Therapeutics for Treatment Access to Migalastat for Specific Adult Patients with Fabry Disease

  • Status: Ongoing, currently recruiting. Physicians may request permission to treat specific patients with migalastat. Patients have amenable GLA mutations, do not meet eligibility criteria for existing migalastat clinical studies, and are unsuitable for or unable to access ERT.
  • Design: Open-label treatment in up to 20 patients for 6 months with renewal every 6 months. More information: www.clinicaltrials.gov: NCT01476163

Fabry Disease Completed Studies

Migalastat Monotherapy: Phase 3 Study 011 (The FACETS, or AT1001-011 Study)

Study of the Effects of Oral Migalastat (AT1001) in Patients with Fabry Disease

  • Status: Completed. In December 2011, the study completed enrollment of 67 patients, who were identified as having amenable GLA mutations and met additional entry criteria.
  • Design: Placebo-controlled, double-blind Phase 3 study of migalastat.
  • More information: www.clinicaltrials.gov: NCT00925301

Migalastat Monotherapy: Phase 3 Study 012 (The ATTRACT or AT1001-012 Study)

Study to Compare the Efficacy and Safety of Oral Migalastat (AT1001) and Enzyme Replacement Therapy in Patients with Fabry Disease

  • Status: Completed. Enrolled more than 50 patients, who were identified as having amenable GLA mutations and had been receiving enzyme replacement therapy (ERT) for at least 12 months.
  • Design: Randomized, open-label, 18-month Phase 3 study investigating the safety and efficacy of migalastat compared to current standard of care ERTs Fabrazyme® (agalsidase beta) or Replagal® (agalsidase alfa) for Fabry disease.
  • More information: www.clinicaltrials.gov: NCT01218659.